Nightstar welcomes $20m

NightstaRx (Nightstar), a fresh spin-out from the University of Oxford, has launched with £12m ($20m) in backing from Syncona, a subsidiary of charity investor the Wellcome Trust.

The company is focused on the development and commercialisation of therapies for degenerative conditions which affect vision. The first retinal dystrophy Nightstar will focus on is a gene therapy for choroideremia, an inherited form of blindness.

The treatment uses a modified virus, AAV.REP1, to correct genetic information of cells in the eye’s retina. In a recent clinical trial published in medical journal The Lancet, the first six patients treated showed improvement in their vision in dim light, with two of the six able to read more lines on the eye chart. Choroideremia affects 1 in 50,000 people, and culminates in blindness in late adulthood. Currently, there is no effective treatment for the condition.

As part of the deal, Chris Hollowood, partner at Syncona, will join Nightstar as its chairman. Syncona also appointed Melanie Lee as the firm’s chief executive.

Robert MacLaren, professor at the Nuffield Laboratory of Ophthalmology at the University of Oxford and lead developer of AAV.REP1 therapy, said: “The initial clinical results for choroideremia gene therapy are very promising and they give us an indication of what this technology can achieve in the future. The Wellcome Trust and the University of Oxford are two of the worlds’ leading biomedical research organisations and they have worked closely together to support the programme. The involvement of Syncona through Nightstar will assist the clinical development, including the manufacture of AAV.REP1 to the stringent requirements needed for regulatory approval, which will expedite patients’ access to the therapy.”

Tom Hockaday, managing director of Oxford’s tech transfer unit Isis Innovation, added: “The £12 million investment in Nightstar represents one of the largest investments in a new academic spin-out in Europe. Isis Innovation is very excited to have worked with Professor MacLaren since 2009 to protect this technology and we look forward to it benefitting patients.”